Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome
<p dir="ltr">Bardet-Biedl syndrome (BBS) and Alström syndrome (AS) are rare autosomal recessive ciliopathies characterized by severe multisystemic involvement, including metabolic, sensory, and developmental impairments. Both conditions result from primary cilia dysfunction, disrupti...
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2025
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| _version_ | 1864513523982270464 |
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| author | Hajar Dauleh (18002500) |
| author2 | Idris Mohammed (751020) Khalid Hussain (110443) |
| author2_role | author author |
| author_facet | Hajar Dauleh (18002500) Idris Mohammed (751020) Khalid Hussain (110443) |
| author_role | author |
| dc.creator.none.fl_str_mv | Hajar Dauleh (18002500) Idris Mohammed (751020) Khalid Hussain (110443) |
| dc.date.none.fl_str_mv | 2025-12-12T15:00:00Z |
| dc.identifier.none.fl_str_mv | 10.1210/jcemcr/luaf266 |
| dc.relation.none.fl_str_mv | https://figshare.com/articles/journal_contribution/Efficacy_of_Semaglutide_in_Pediatric_Patients_With_Bardet-Biedl_Syndrome_and_Alstr_m_Syndrome/31239460 |
| dc.rights.none.fl_str_mv | CC BY 4.0 info:eu-repo/semantics/openAccess |
| dc.subject.none.fl_str_mv | Biomedical and clinical sciences Medical biochemistry and metabolomics Paediatrics Alström syndrome Bardet-Biedl syndrome semaglutide obesity GLP-1 agonist |
| dc.title.none.fl_str_mv | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| dc.type.none.fl_str_mv | Text Journal contribution info:eu-repo/semantics/publishedVersion text contribution to journal |
| description | <p dir="ltr">Bardet-Biedl syndrome (BBS) and Alström syndrome (AS) are rare autosomal recessive ciliopathies characterized by severe multisystemic involvement, including metabolic, sensory, and developmental impairments. Both conditions result from primary cilia dysfunction, disrupting pathways such as leptin and insulin signaling, which leads to obesity and insulin resistance. While setmelanotide, a melanocortin-4 receptor agonist, may be an effective therapy for obesity in these conditions (especially BBS), its cost limits accessibility for many patients. We describe two pediatric cases (one with BBS and another with AS) demonstrating significant metabolic improvements with semaglutide, a glucagon-like peptide-1 receptor agonist. These observations suggest that some cases of BBS and AS may be treated with semaglutide.</p><h2 dir="ltr">Other Information</h2><p dir="ltr">Published in: JCEM Case Reports<br>License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1210/jcemcr/luaf266" target="_blank">https://dx.doi.org/10.1210/jcemcr/luaf266</a></p> |
| eu_rights_str_mv | openAccess |
| id | Manara2_657b2c6cb98ba935502f5762b3c01a54 |
| identifier_str_mv | 10.1210/jcemcr/luaf266 |
| network_acronym_str | Manara2 |
| network_name_str | Manara2 |
| oai_identifier_str | oai:figshare.com:article/31239460 |
| publishDate | 2025 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| rights_invalid_str_mv | CC BY 4.0 |
| spelling | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström SyndromeHajar Dauleh (18002500)Idris Mohammed (751020)Khalid Hussain (110443)Biomedical and clinical sciencesMedical biochemistry and metabolomicsPaediatricsAlström syndromeBardet-Biedl syndromesemaglutideobesityGLP-1 agonist<p dir="ltr">Bardet-Biedl syndrome (BBS) and Alström syndrome (AS) are rare autosomal recessive ciliopathies characterized by severe multisystemic involvement, including metabolic, sensory, and developmental impairments. Both conditions result from primary cilia dysfunction, disrupting pathways such as leptin and insulin signaling, which leads to obesity and insulin resistance. While setmelanotide, a melanocortin-4 receptor agonist, may be an effective therapy for obesity in these conditions (especially BBS), its cost limits accessibility for many patients. We describe two pediatric cases (one with BBS and another with AS) demonstrating significant metabolic improvements with semaglutide, a glucagon-like peptide-1 receptor agonist. These observations suggest that some cases of BBS and AS may be treated with semaglutide.</p><h2 dir="ltr">Other Information</h2><p dir="ltr">Published in: JCEM Case Reports<br>License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1210/jcemcr/luaf266" target="_blank">https://dx.doi.org/10.1210/jcemcr/luaf266</a></p>2025-12-12T15:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1210/jcemcr/luaf266https://figshare.com/articles/journal_contribution/Efficacy_of_Semaglutide_in_Pediatric_Patients_With_Bardet-Biedl_Syndrome_and_Alstr_m_Syndrome/31239460CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/312394602025-12-12T15:00:00Z |
| spellingShingle | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome Hajar Dauleh (18002500) Biomedical and clinical sciences Medical biochemistry and metabolomics Paediatrics Alström syndrome Bardet-Biedl syndrome semaglutide obesity GLP-1 agonist |
| status_str | publishedVersion |
| title | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| title_full | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| title_fullStr | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| title_full_unstemmed | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| title_short | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| title_sort | Efficacy of Semaglutide in Pediatric Patients With Bardet-Biedl Syndrome and Alström Syndrome |
| topic | Biomedical and clinical sciences Medical biochemistry and metabolomics Paediatrics Alström syndrome Bardet-Biedl syndrome semaglutide obesity GLP-1 agonist |